MRC Funding for Translational Research

Transcription

1 MRC Funding for Translational Research Dr Tom Foulkes May 2011

2 Translational research - importance to the MRC Central to delivery of MRC s mission Recent favourable CSR settlement translational work cited as critical due to the potential for health and economic impact Significant budget to support translational work increasing Features heavily in MRC s Delivery Plan including commitment to spend 50m p.a. by 2014/15 on managed programmes Exciting opportunities to exploit findings from basic science Many clinical problems (e.g. obesity) becoming increasingly tractable Investment in basic research must be maintained to ensure pipeline continues Translational work so far regarded as very successful We are keen to receive high quality applications to all schemes (particularly DPFS and DCS)

3 Oxford as a centre for translational research Very strong basic and clinical science Close links to hospital Wide range of expertise project management, pre-clinical and clinical research, many academic areas One DCS already, 3 DPFS MRC Units & Centres

4 MRC Translational Funding Schemes Developmental Pathway Funding Scheme (DPFS)* Preclinical development multiple DPFS if required Developmental Clinical Studies (DCS) Phase I-II Translational Stem Cell Research Committee (TSCRC) MRC-NIHR Efficacy, Mechanism and Evaluation (EME) Phase IIb-III * Emerging concept DPFS programme grants

5 Specialist MRC Translational Funding Schemes Experimental Medicine for Mental Health Workshop stage calls to be announced next year cineformentalhealth

6 Translational Funding Since Inception Last 12 months Scheme Number Value Number Value Leading Therapeutic Areas Since Inception Leading Modalities DPFS 97 c. 33m 21 c. 12m DCS 13 c. 19m 10 c. 14m TSCRC 17 c. 11m 6 c. 4m Cancer, Infection, Neurological Cardiovascular, Infection, Stroke Eye, Oral and GI, Inflammatory and Immune System, Musculoskeletal Small Molecule, Diagnostic Non-Imaging, Protein/Peptide Protein/Peptide, Small Molecule, Vaccine Regenerative Medicine TOTAL 127 c. 64m 37 c. 30m

7 Developmental Clinical Studies Phase I and II safety and proof of concept in man Can be new intervention, or change in use of existing intervention Can include regulatory toxicology, manufacture to GMP etc. if required for trial to be undertaken Modality: small molecules, cells, peptides, antibodies, vaccines, devices, diagnostics, psychotherapeutics etc. Can include multiple phases, or end point could be another DCS Not included: Phase III studies (funded by EME scheme) Principal focus to understand aetiology Animal/non-human studies (except where required for regulatory reasons) Stem cells (assessed by separate MRC Panel - TSCRC)

8 Milestones Overall purpose to allow the MRC to take risks Allows investment in high-risk, high-reward projects (remaining funds can be recovered if project fails) Provides confidence in project management Permits funding of contingent projects (i.e. part 2 necessary/possible only if part 1 works) Early discussion best if problems encountered can agree changes to plan Projects will and have been closed if milestones not met This does not imply failure by the applicants we accept that there is inherent risk in this type of research

9 Industrial Collaboration MRC Industry Collaboration Award (all funding schemes) 2 categories: Pre-negotiated foreground IP Minimum 50% industry contribution No pre-negotiated position (first refusal OK) No minimum contribution Additional form ensures MRC meets EU state-aid regulations

10 DCS - How to be successful Sufficient clinical need Background proof of concept in model systems Appropriate design (trial design expertise) Relevant endpoints Statistics/powering (collaborate) Deliverability esp. realistic recruitment plans (with evidence if possible) Rigorous milestones (more on this later) Clear plans for progression to clinic Consider inclusion/exclusion criteria Realistic costing (DCS awards made 300k - 3m) Talk to us if questions

11 Case Study: PYY for treatment of obesity Batterham et al NEJM

12 Case Study: PYY for treatment of obesity Clinical need: Obesity is a significant public health concern, and current treatments are either invasive or insufficiently efficacious Intervention: PYY analogue (new) Rationale: PYY is an endogenous peptide involved in the regulation of appetite. PoC in animal models established Project: To establish safety, tolerability and pharmacokinetics of PYY analogue in Phase I clinical study Starting point: Manufacture of product to GMP quality End point: 2 studies on toxicology in man, pharmacokinetic and preliminary efficacy data, all in healthy volunteers Cost: 2.8m (FEC)

13 Case Study: Sodium nitrite for ischaemiareperfusion injury in myocardial infarction Lundberg et al. 2008

14 Case Study: Sodium nitrite for ischaemiareperfusion injury in myocardial infarction Clinical need: Loss of myocardium following AMI often results in chronic heart failure Intervention: Nitrite (endogenous anion) Rationale: Mechanistic (complex) + animal PoC Project: Single efficacy study (first time tried in man for this indication) Starting point: Healthy volunteer study already completed End point: Efficacy study in patients completed Cost: 1.5m

15 Follow-on Funding Routes Can partner with industry at any stage Rationale for staying in publicly-funded schemes justification requirements increase as project progresses If staying in the publicly-funded domain: Multiple DPFS (if further development required) DCS EME Funds available (2009/10): DPFS + DCS 30m (and increasing)

16 How to apply Online via JeS Applications taken to appropriate Research Boards Distinct Case for Support structure guidance online Additional requirements: Milestones form Gantt chart Letter of support from TTO

17 Sources of help JeS Helpdesk: General advice through MRC staff and through translators University clinical trials support, research office etc. Iteration for appropriate applications Any other ideas?